US10889607B2ActiveUtilityA1
Sialic acid analogs
Assignee: ULTRAGENYX PHARMACEUTICAL INCPriority: Oct 24, 2011Filed: Sep 16, 2019Granted: Jan 12, 2021
Est. expiryOct 24, 2031(~5.3 yrs left)· nominal 20-yr term from priority
C07D 493/08C07H 7/027C07H 13/10C07H 13/04C07H 7/02A61P 21/00
73
PatentIndex Score
0
Cited by
97
References
20
Claims
Abstract
The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
Claims
exact text as granted — not AI-modifiedWhat is claimed is:
1. A compound selected from the group consisting of:
2. The compound of claim 1 , wherein the compound is:
3. The compound of claim 1 , wherein the compound is:
4. The compound of claim 1 , wherein the compound is:
5. The compound of claim 1 , wherein the compound is:
6. The compound of claim 1 , wherein the compound is:
7. The compound of claim 1 , wherein the compound is:
8. A pharmaceutical composition comprising a compound selected from the group consisting of:
or a pharmaceutically acceptable salt or solvate thereof, and a pharmaceutically acceptable carrier.
9. The pharmaceutical composition of claim 8 , comprising the compound:
10. The pharmaceutical composition of claim 8 , comprising the compound:
11. The pharmaceutical composition of claim 8 , comprising the compound:
12. The pharmaceutical composition of claim 8 , comprising the compound:
13. The pharmaceutical composition of claim 8 , comprising the compound:
14. The pharmaceutical composition of claim 8 , comprising the compound:
15. A sustained release pharmaceutical composition comprising a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof, wherein:
a) the release of the compound is over a period of about 4 hours to about 24 hours; and/or
b) the pharmacological effect from the compound lasts about four hours to about 24 hours upon administration of the composition.
16. A sustained release pharmaceutical composition comprising a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof,
wherein the composition, upon administration, provides a therapeutically effective amount of the compound for about 4 hours to about 24 hours.
17. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering an effective amount of a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof.
18. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof wherein upon administration, the compound, or a pharmaceutically acceptable salt or solvate thereof, continuously provides a therapeutically effective amount of sialic acid for about 4 hours to about 24 hours.
19. The method of claim 17 , wherein the sialic acid deficiency is myopathy caused by sialic acid deficiency.
20. The method of claim 19 , wherein the myopathy caused by sialic acid deficiency is Hereditary Inclusion Body Myopathy (HIBM), Nonaka myopathy, or Distal Myopathy with Rimmed Vacuoles (DMRV).Cited by (0)
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